Objective: To investigate the clinical features of transplant-associated thrombotic microangiopathy (TA-TMA) and the prognostic value of different prognostic risk models for TA-TMA.
Methods: The clinical characteristics of 32 TA-TMA patients diagnosed at the First Medical Center of the PLA General Hospital from January 2018 to February 2022 in terms of short-term prognosis and influencing factors were retrospectively analyzed. In addition, the risk population composition ratio, treatment response, and overall survival between the BATAP risk model and the TMA index model were compared, as well as the efficacy of two prognostic risk models for predicting death in patients with TA-TMA.
Results: Independent risk factors affecting the short-term prognosis of TA-TMA include III-IV aGVHD prior to TA-TMA diagnosis (P=0.001), renal or neurological dysfunction (P=0.006), and Hb<70 g/L (P=0.043). In the TMA index model, treatment response was worst in the high-risk group (P=0.008), while there was no significant difference in treatment response between different risk groups in the BATAP model (P=0.105). In the BATAP model, there was a statistically significant difference in the OS between the three groups of low risk, intermediate risk, and high risk (87.5% vs 61.1% vs 16.7%, χ2＝6.7, P=0.014). In the TMA index model, there was a statistically significant difference in the OS between the three groups of low risk, intermediate risk, and high risk (77.8% vs 45.5% vs 0.0%, χ2＝7.3, P=0.017). The area under the ROC curve (AUC) of the TMA index model was 0.745 (95%CI: 0.56-0.88, P<0.05), and the AUC of the BATAP model was 0.743 (95%CI: 0.56-0.88, P<0.05), indicating that both prognostic risk models have good predictive value.
Conclusion: The short-term prognosis of TA-TMA patients might be accurately determined using both the BATAP model and the TMA index model. When predicting the efficacy of TA-TMA in different risk groups, the TMA index model may perform better than the BATAP model.
题目: 基于不同预后风险模型对移植相关血栓性微血管病的疗效及预后分析.
目的: 研究移植相关血栓性微血管病（TA-TMA）临床特征及不同预后风险模型对TA-TMA的预后价值.
方法: 选择2018年1月至2022年2月在解放军总医院第一医学中心诊断的32例TA-TMA患者为研究对象，回顾性分析TA-TMA患者短期预后及相关影响因素等临床特征，BATAP风险模型和TMA指数模型中TA-TMA风险人群构成比、治疗反应、生存情况，以及两种预后风险模型对TA-TMA死亡预测效能进行比较.
结果: TA-TMA诊断前III-IV度aGVHD（P=0.001），肾脏或神经系统损伤（P=0.006），诊断时Hb＜70 g/L（P=0.043）是影响TA-TMA短期预后的独立危险因素。TMA指数模型中，高危组治疗反应最差（P=0.008）；BATAP模型中不同危险程度组间治疗反应无明显差异（P=0.105）。在BATAP模型中，低危组、中危组、高危组的OS率分别为87.5%、61.1%和16.7%，差异有统计学意义（χ2＝6.7，P=0.014）；在TMA指数模型中，低危组、中危组、高危组的OS率分别为77.8%、45.5%和0.0%，差异有统计学意义（χ2＝7.3，P<0.0001）。TMA指数模型ROC曲线下面积（AUC）为0.745（95%CI： 0.56-0.88，P＜0.05），BATAP模型AUC为0.743（95%CI： 0.56-0.88，P＜0.05），提示两种预后风险模型均具有良好的预测价值.
结论: BATAP模型和TMA指数模型均能较好的评估TA-TMA患者短期预后，TMA指数模型在预测TA-TMA不同风险人群的治疗反应方面可能优于BATAP风险模型.
Keywords: allogeneic hematopoietic stem cell transplantation; prognostic risk models; thrombotic microangiopathy; transplant-associated TMA.